Please provide your email address to receive an email when new articles are posted on . Wainua has received regulatory approval for polyneuropathy of hereditary transthyretin-mediated amyloidosis.

If you or a loved one are living with hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM), you are likely well-acquainted with the challenges of heart health. You know the fatigue, the ...

The outlook for hATTR-PN can vary depending on the genetic mutation involved and how the condition first presents. Newer treatments are helping improve outcomes. hATTR is a rare, hereditary disease. A ...

Hereditary transthyretin amyloidosis (hATTR) is a rare inherited disorder that gets worse over time. It often affects the nerves, heart, liver, and kidneys, but it can also affect other organs.

Intellia Therapeutics stock surged Tuesday after the FDA aid it would allow the company to restart a gene-editing study.

FDA has lifted the clinical hold on Intellia Therapeutics' MAGNITUDE-2 Phase 3 trial in hereditary transthyretin amyloidosis with polyneuropathy. The decision allows the late stage study of Intellia's ...

A gene-editing therapy led to durable changes in serum transthyretin levels among people with hereditary transthyretin amyloidosis with polyneuropathy. Polyneuropathy stages and disability scores were ...

CARLSBAD, Calif., Dec. 21, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (IONS) (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has approved Ionis and AstraZeneca's ...

WILMINGTON, Del.--(BUSINESS WIRE)-- Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed ...

"Our data support the use of sNfL in screening TTRv carriers and in monitoring disease progression and effect of treatment in ATTRv amyloidosis patients…Larger studies are warranted to confirm and ...

WILMINGTON, Del.--(BUSINESS WIRE)-- Eplontersen has been granted Orphan Drug Designation (ODD) in the US by the Food and Drug Administration (FDA) for the treatment of transthyretin-mediated ...